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DT-216P1

Development stage: Discontinued
Lead developer: Design Therapeutics
Modality: Small Molecules
Administration: Intravenous

Overview

DT-216P1 is a small molecule gene modulator developed as part of Design Therapeutics' GeneTAC platform for the treatment of Friedreich ataxia. It is designed to increase the expression of frataxin, a mitochondrial protein deficient in patients with Friedreich ataxia. DT-216P1 acts as a genetic transcription modulator and frataxin expression stimulant. It was evaluated in early-phase clinical trials for its safety, pharmacokinetics, and pharmacodynamics. Development of DT-216P1 was discontinued due to issues including injection site thrombophlebitis, and improved molecules such as DT-216P2 have since advanced in the pipeline[1][4][5][11][12].

Other names: DT-216DT216DT 216DT-216P1DT216P1DT 216P1

Targets

FXN GAA repeat (Frataxin GAA repeat expansion)

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